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Midostaurin · Rydapt, CGP-41251

Small molecule · Inhibitor of FLT3, KIT and PDGFRA · developed by Novartis

Approved FDA, Apr 2017
1 Registered trials

What is Midostaurin?

Midostaurin is a small-molecule kinase inhibitor that blocks FLT3, KIT, PDGFRα/β, and protein kinase C, inhibiting cell proliferation and inducing apoptosis in leukemic and mast cells.

Summary written with AI from the sources listed at the end of this page.

At a glance

Also known as
Rydapt, CGP-41251, NSC-656576
Drug type
Small molecule
Targets
FLT3, KIT, PDGFRA, PDGFRB, protein kinase C (PRKCA), VEGFR2 (KDR) (secondary target)
Mechanism
Inhibitor of FLT3, KIT and PDGFRA
Pathways
cell cycle disruption, apoptosis, angiogenesis inhibition
Therapy area
Oncology
Developer
Novartis
In trials of
SNDX
FDA status
Approved as Rydapt (April 28, 2017), NDA207997 · FDA label
UNII
ID912S5VON

✅ FDA-approved uses

From the indications section of the FDA label (version of Jun 8, 2026). Read the full label for the exact wording, limitations and safety information.

🧪 Clinical trials

TrialPhaseConditionStatusSponsor
NCT06313437 Phase 1 Acute Myeloid Leukemia Recruiting SNDX

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Acute Myeloid LeukemiaAMLAML With Gene MutationsAML, AdultLeukemia

Other drugs acting on FLT3 / KIT / PDGFRA / PDGFRB / PRKCA

Gilteritinib FDA approved Nintedanib FDA approved Pexidartinib FDA approved Regorafenib FDA approved Sunitinib FDA approved 89Zr-DFOsq-olaratumab Phase 1 Barzolvolimab Phase 3 Bezuclastinib Phase 3 BMF-500 Phase 1 Briquilimab Phase 1/2 CDX 0158 Phase 1 CDX-301 Phase 2

Sources

Updated Oct 3, 2026.

Data: FDA (Drugs@FDA, Purple Book, DailyMed), NIH (NCI Thesaurus, GSRS, RxNorm), ClinicalTrials.gov • Informational purposes only, not medical or investment advice. • All drugs A–Z

This data is for informational purposes only, not investment advice. BioRadar does not provide buy/sell recommendations. Past performance does not guarantee future results. Always do your own due diligence.